Palbociclib and endocrine therapy in heavily pretreated hormone receptor-positive HER2-negative advanced breast cancer: the UK Compassionate Access Programme experience
Palbociclib is approved in 1st line for hormone receptor (HR)-positive HER2-negative advanced breast cancer (ABC). A Compassionate Access Programme previously allowed patients to receive it in 4th line. However, Palbociclib has not been specifically tested in this population. We aimed to determine the safety and efficacy profile of Palbociclib within the Programme across ten institutions in the United Kingdom.
We retrospectively identified HR-positive HER2-negative ABC patients on the Programme between December 2015 and September 2017. Demographics, disease characteristics, prior treatments, blood tests, toxicities, treatment delays and responses were recorded. Simple statistics, Fisher’s exact test, χ2 method and Cox regression were used.
118 patients identified had a median age of 59. 82.2% were postmenopausal and 92.4% performance status 0–1. 81.4% had visceral involvement and 6.8% bone-only disease after a median of 5 prior treatments and 3 prior chemotherapies. Clinical benefit rate was 47.5%, overall response rate 15.8%, median PFS 4.5 months and median OS 15.8 months. Longer progression-free survival on prior endocrine therapy was a predictor of longer PFS and OS. 89.7% developed neutropenia (grade ≥ 3 in 56.8%). 5.1% experienced febrile neutropenia. 48.3% had dose reductions and 3.4% discontinued Palbociclib following toxicity. No statistically significant difference in grade ≥ 3 neutropenia was observed according to metastatic sites nor previous treatments.
This is the most extensive analysis of palbociclib in ≥ 4th-line setting. Clinical benefit was confirmed particularly for endocrine-sensitive, predominantly bony disease and in earlier lines of treatment. Safety was similar to PALOMA trials with higher febrile neutropenia rate.
KeywordsBreast cancer ER-positive Palbociclib Advanced stage Pretreated
The authors wish to acknowledge the support of Pfizer in providing a list of centres enrolling patients within the Compassionate Access Programme. Prof Johnston, Dr Ring, Dr Battisti and Dr Kingston wish to acknowledge the support of The Royal Marsden NIHR Biomedical Research Centre for Cancer.
Conflict of interest
Prof Johnston has received consulting and advisory board fees from Eli Lilly, AstraZeneca and Puma Biotechnology, speaker fees from Pfizer and Novartis and research funding from Pfizer. Dr Ring has received advisory board fees from Novartis, Pfizer and Lilly and speaker fees from Novartis and Pfizer. Dr Roylance has received an institutional educational grant from Pfizer and honoraria from Pfizer for advisory board and educational meetings. Dr Waters has received speaker and advisory fees from Genomic Health International, Novartis UK and Eisai Ltd. Dr Battisti, Dr Kingston, Dr Harper, Dr King, Dr Denton, Dr Sita-Lumsden, Dr Rehman, Dr Stavraka, Dr Kristeleit, Prof Sawyer, Dr Houghton, Prof Davidson, Dr Howell, Ms Choy, Dr Fharat and Mr Mohammed have no conflict of interest.
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